EP1379134A4 - Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions - Google Patents

Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions

Info

Publication number
EP1379134A4
EP1379134A4 EP02719279A EP02719279A EP1379134A4 EP 1379134 A4 EP1379134 A4 EP 1379134A4 EP 02719279 A EP02719279 A EP 02719279A EP 02719279 A EP02719279 A EP 02719279A EP 1379134 A4 EP1379134 A4 EP 1379134A4
Authority
EP
European Patent Office
Prior art keywords
gland
virions
secretory
recombinant adeno
retroductal
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP02719279A
Other languages
German (de)
English (en)
Other versions
EP1379134A1 (fr
Inventor
Alan Mcclelland
Roland Scollay
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Avigen Inc
Genteric Inc
Original Assignee
Avigen Inc
Genteric Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Avigen Inc, Genteric Inc filed Critical Avigen Inc
Publication of EP1379134A1 publication Critical patent/EP1379134A1/fr
Publication of EP1379134A4 publication Critical patent/EP1379134A4/fr
Withdrawn legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

l'invention concerne des procédés d'introduction de virions de virus recombiné adéno-associé (rAAV) dans une cellule ou dans des cellules d'une glande sécrétrice. Des virions AAV recombinés contenant un gène hétérologue sont introduits dans le conduit d'une glande sécrétrice résultant en une transduction d'une ou plusieurs cellules de glande sécrétrice. Lorsqu'une cellule de glande sécrétrice est transduite par le virion rAAV, le gène hétérologue est exprimé et le produit d'expression est sécrété. Des exemples de glandes sécrétrices sont le foie, la glande submandibulaire, la glande parotide, et la glande sublinguale. L'utilisation des procédés de l'invention permet d'obtenir des taux thérapeutiques de protéine. L'invention concerne aussi des procédés de traitement de l'hémophilie.
EP02719279A 2001-03-14 2002-03-14 Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions Withdrawn EP1379134A4 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US27590801P 2001-03-14 2001-03-14
US275908P 2001-03-14
PCT/US2002/008350 WO2002071843A1 (fr) 2001-03-14 2002-03-14 Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions

Publications (2)

Publication Number Publication Date
EP1379134A1 EP1379134A1 (fr) 2004-01-14
EP1379134A4 true EP1379134A4 (fr) 2006-04-05

Family

ID=23054332

Family Applications (1)

Application Number Title Priority Date Filing Date
EP02719279A Withdrawn EP1379134A4 (fr) 2001-03-14 2002-03-14 Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions

Country Status (5)

Country Link
US (1) US20030147853A1 (fr)
EP (1) EP1379134A4 (fr)
JP (1) JP2004532822A (fr)
CA (1) CA2441454A1 (fr)
WO (1) WO2002071843A1 (fr)

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WO2001096587A2 (fr) * 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia Procede d'administration de virions recombinants de virus associes aux adenovirus a des etres humains ayant ete exposes a ce type de virus
US20040009151A1 (en) * 2002-04-04 2004-01-15 Kay Mark A. Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal
WO2004108922A2 (fr) * 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Procedes et compositions pour abaisser le niveaux totaux de cholesterol et traitement des maladies cardiaques
JP2008521575A (ja) * 2004-12-01 2008-06-26 ジェンザイム・コーポレイション 肝臓を標的とした遺伝物質の送達方法
US20100291160A1 (en) * 2009-05-13 2010-11-18 Carver David R Pharmaceutical system for trans-membrane delivery
BR112015003694A2 (pt) 2012-08-29 2017-07-04 Unitract Syringe Pty Ltd mecanismo de acionamento de entrega controlada e bomba de entrega de fármaco com entrega de fármaco controlada
JP2017509328A (ja) 2014-03-21 2017-04-06 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー ヌクレアーゼを使用しないゲノム編集
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
CL2014002410A1 (es) * 2014-09-12 2014-11-14 Univ Concepcion Método para la producción de proteínas recombinantes en glándula mamaria de mamíferos mediante la transformación del epítelio glandular mamario con vectores adenoasociados.
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
ES2878451T3 (es) 2014-11-14 2021-11-18 Voyager Therapeutics Inc Polinucleótidos moduladores
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016094783A1 (fr) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
RU2762257C2 (ru) 2016-04-15 2021-12-17 Зе Трастис Оф Зе Юниверсити Оф Пенсильвания Генная терапия для лечения гемофилии a
WO2017189964A2 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
EP3448874A4 (fr) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
CA3035522A1 (fr) 2016-08-30 2018-03-08 The Regents Of The University Of California Procedes de ciblage et d'administration biomedicaux, et dispositifs et systemes pour la mise en ƒuvre de ceux-ci
WO2018144709A2 (fr) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Thérapie génique pour le traitement de citrullinémie
WO2018204803A1 (fr) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la maladie de huntington
WO2018204786A1 (fr) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la sclérose latérale amyotrophique (sla)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP4124658A3 (fr) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CA3173768A1 (fr) 2020-10-13 2022-04-21 Brian Furmanski Constructions de vecteurs viraux pour l'apport d'acides nucleiques codant pour des cytokines et leurs utilisations pour le traitement du cancer
WO2024054993A1 (fr) 2022-09-09 2024-03-14 Kriya Therapeutics, Inc. Constructions de vecteur pour l'administration d'acides nucléiques codant pour des anticorps anti-ctla4 thérapeutiques et leurs procédés d'utilisation

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WO2000023116A1 (fr) * 1998-10-20 2000-04-27 Avigen, Inc. Vecteurs adeno-associes pour l'expression du facteur viii par des cellules cibles
WO2001096587A2 (fr) * 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia Procede d'administration de virions recombinants de virus associes aux adenovirus a des etres humains ayant ete exposes a ce type de virus

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US5837693A (en) * 1995-03-24 1998-11-17 The Regents Of The University Of California Intravenous hormone polypeptide delivery by salivary gland expression
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WO2000023116A1 (fr) * 1998-10-20 2000-04-27 Avigen, Inc. Vecteurs adeno-associes pour l'expression du facteur viii par des cellules cibles
WO2001096587A2 (fr) * 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia Procede d'administration de virions recombinants de virus associes aux adenovirus a des etres humains ayant ete exposes a ce type de virus

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NAKAI H. ET AL.: "A proposed rAAV liver -directed clinical trial for hemophilia B.", BLOOD, vol. 11, no. 1, 16 November 2000 (2000-11-16), pages 798A - 799A, XP009061145 *
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SNYDER R O ET AL: "Correction of hemophilia B in canine and murine models using adeno-associated viral vectors", NATURE MEDICINE, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 5, no. 1, January 1999 (1999-01-01), pages 64 - 70, XP002190250, ISSN: 1078-8956 *

Also Published As

Publication number Publication date
WO2002071843A1 (fr) 2002-09-19
US20030147853A1 (en) 2003-08-07
JP2004532822A (ja) 2004-10-28
EP1379134A1 (fr) 2004-01-14
CA2441454A1 (fr) 2002-09-19

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